Although myeloablative conditioning (MAC) before haploidentical donor transplant (HIDT) with post-transplant cyclophosphamide is being increasingly used, the optimal preparative regimen remains unclear. In our initial trial, the feasibility of HIDT following a MAC preparative regimen using fludarabine and 12 Gy of total-body irradiation was demonstrated in 30 patients. We now present long-term outcome results, including an additional 52 patients, now with 47 months (16 to 96) median follow-up. Median patient age was 42 (19 to 61) years. The most common diagnoses were acute myelogenous leukemia (51%) and acute lymphoblastic leukemia (33%), and 39% had a high/very high disease risk index (DRI). Engraftment was universal with no cases of primary or secondary graft failure. Grade 3 to 4 acute graft-versus-host disease (GVHD) and moderate to severe chronic GVHD occurred in 17% and 23%, respectively. Nonrelapse mortality (NRM) was 7% at 1 year and 13% at 4 years. Estimated 4-year overall survival (OS), disease-free survival, and cumulative incidence of relapse (CIR) were 67%, 60%, and 27%, respectively. CIR was significantly higher in patients with high/very high- versus low/intermediate-risk DRI (38% versus 20%, P= .032), which led to inferior 4-year OS (50% versus 77%, P = .001). Median time to systemic immunosuppressive therapy (IST) discontinuation was 7.8 months, with 84% of patients off IST at 2 years post-transplant. Current GHVD-free, relapse-free survival (CGRFS) at 2, 3, and 4 years was 60%, 57%, and 60%, respectively. This approach to MAC HIDT results in universal engraftment; low rates of NRM, infection, and clinically significant GVHD; and relatively rapid IST discontinuation, resulting in high rates of CGRFS and survival.

中文翻译：

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消融单倍体移植前的氟达拉滨和全身照射调理：长期安全性和有效性。

尽管越来越多地采用异体供体移植（HIDT）和移植后环磷酰胺进行骨髓分离条件（MAC），但尚不清楚最佳的制备方案。在我们的初步试验中，在30例患者中证明了使用氟达拉滨和12 Gy全身照射进行MAC制备方案后进行HIDT的可行性。我们现在提供长期结果，包括另外52例患者，现在进行了47个月（16到96）的中位随访。患者中位年龄为42岁（19至61岁）。最常见的诊断是急性骨髓性白血病（51％）和急性淋巴细胞性白血病（33％），39％的疾病风险指数（DRI）高/非常高。移植是普遍的，没有原发性或继发性移植失败的情况。3-4级急性移植物抗宿主病（GVHD）和中重度慢性GVHD分别发生在17％和23％。非复发死亡率（NRM）在1年时为7％，在4年时为13％。估计的4年总生存期（OS），无病生存期和累积复发率（CIR）分别为67％，60％和27％。高/极高风险与低/中风险DRI患者的CIR显着较高（38％对20％，P = .032），导致4年OS降低（50％对77％，P =。 001）。全身免疫抑制治疗（IST）中断的中位时间为7.8个月，其中84％的患者在移植后2年停用IST。目前在2年，3年和4年时无GHVD，无复发生存（CGRFS）分别为60％，57％和60％。MAC HIDT的这种方法导致普遍植入。NRM率低，感染，具有临床意义的GVHD；以及相对快速的IST停药，导致CGRFS和生存率较高。